{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,5]],"date-time":"2026-11-05T03:22:01Z","timestamp":1793848921908,"version":"4.6.1"},"reference-count":31,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2017,4,25]],"date-time":"2017-04-25T00:00:00Z","timestamp":1493078400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Alzheimer\u2019s Disease"],"published-print":{"date-parts":[[2017,5,11]]},"abstract":"<jats:p>Background: ELND005 ( scyllo-Inositol; cyclohexane-1,2,3,4,5,6-hexol) has been evaluated as a potential disease-modifying treatment for Alzheimer\u2019s disease (AD). Individuals with Down syndrome (DS) have an increased risk for developing AD dementia.<\/jats:p>\n                  <jats:p>Objective: To evaluate the safety and tolerability of ELND005 and to determine its pharmacokinetics (PK) and relationship between PK parameters, safety outcome measures, and exploratory efficacy outcome measures in young adults with DS without dementia.<\/jats:p>\n                  <jats:p>Methods: This was a prospective, randomized, double-blind, placebo-controlled, parallel-group, three-arm, multicenter Phase II study of the safety and pharmacokinetics of ELND005 administered orally for 4 weeks (ClinicalTrials.gov NCT01791725). Participants who met study eligibility criteria were randomly assigned in a 2\u200a:\u200a1:1 ratio to receive ELND005 at either 250\u200amg twice daily (BID) or 250\u200amg once daily (QD) or matching placebo for 4 weeks.<\/jats:p>\n                  <jats:p>Results: There were no apparent treatment group-related trends on cognitive or behavioral measures and there were no SAEs and no deaths in the study. Overall, mean changes from baseline in clinical laboratory parameters, vital sign measurements, electrocardiogram results, and other physical findings were unremarkable. ELND005 accumulation averaged approximately 2-fold with QD dosing, and 3- to 4-fold with BID dosing.<\/jats:p>\n                  <jats:p>Conclusion: Overall, treatment of adults with DS with ELND005 at both doses was well tolerated, achieved measurable blood levels and demonstrated no safety findings. Further studies will be needed to test efficacy.<\/jats:p>","DOI":"10.3233\/jad-160965","type":"journal-article","created":{"date-parts":[[2017,4,25]],"date-time":"2017-04-25T11:04:01Z","timestamp":1493118241000},"page":"401-411","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":38,"title":["A Randomized, Double-Blind, Placebo-Controlled, Phase II Study of Oral ELND005 (\n                    <i>scyllo<\/i>\n                    -Inositol) in Young Adults with Down Syndrome without Dementia"],"prefix":"10.1177","volume":"58","author":[{"given":"Michael S.","family":"Rafii","sequence":"first","affiliation":[{"name":"Alzheimer\u2019s Therapeutic Research Institute (ATRI) at University of Southern California, San Diego, CA, USA"},{"name":"Department of Neurosciences, University of California, San Diego, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian G.","family":"Skotko","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Down Syndrome Program, Division of Medical Genetics, Massachusetts General Hospital, Boston, MA, USA"},{"name":"Harvard Medical School, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary Ellen","family":"McDonough","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Down Syndrome Program, Division of Medical Genetics, Massachusetts General Hospital, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Margaret","family":"Pulsifer","sequence":"additional","affiliation":[{"name":"Harvard Medical School, Boston, MA, USA"},{"name":"Department of Psychiatry, Psychology Assessment Center, Massachusetts General Hospital, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Casey","family":"Evans","sequence":"additional","affiliation":[{"name":"Department of Psychiatry, Psychology Assessment Center, Massachusetts General Hospital, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Doran","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of California, Irvine, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabriela","family":"Muranevici","sequence":"additional","affiliation":[{"name":"Department of Neurosciences, University of California, San Diego, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick","family":"Kesslak","sequence":"additional","affiliation":[{"name":"ACADIA Pharmaceuticals, San Diego, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan","family":"Abushakra","sequence":"additional","affiliation":[{"name":"Alzheon, Inc., Framingham, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ira T.","family":"Lott","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of California, Irvine, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"for the ELND005-DS Study 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